new treatment

Clinical trial testing experimental enzyme replacement drug in people with Pompe disease who are at least 13 years old

posted on November 8, 2011 - 10:08am

Update (March 27, 2013): BioMarin announced March 19, 2013, that it plans to move to a phase 2/3 trial of this drug for late-onset Pompe diseae, pending a review by regulatory authorities. See Pompe disease: Late-onset treatment moves forward.